>>www.pei.de

The Innovation Office

at the Paul-Ehrlich-Institut

Bettina Ziegele, M.A., Head Innovation Office

SME-Workshop„Focus on Scientific and Regulatory Advice“, 26 May 2011

Innovation Office

FrameworkLegal BasisKinds of Advice and FocusServicesStatisticsFuture ProjectsChallenges

OverviewOverview

Innovation Office

February 2009 – August 2009: Setting of formal requirements together with the German Federal Ministry of Health

August 2009 – October 2009: Preparations for implementing the Innovation Office

Start of work November 2009: Press release, presentation at the website of the PEI

December 2009: Opening workshop at the PEI

Implementation of the Innovation OfficeImplementation of the Innovation Office

Innovation Office

• A marketing authorisation is required for industrially produced ATMP.

• Those ATMP having legally been on the national market by 30 December 2008 need a marketing authorisation after a transition period:• gene and cell therapy products starting 30 December 2011• TEPs starting 30 December 2012

• Marketing authorisation is provided via the centralised procedure co-ordinated by the European Medicines Agency (EMA).

• A full dossier (Directive 2009/120/EC incl. Annex I, Part IV) will be reviewed by experts in the• Committee for Advanced Therapies (CAT; PEI membership).

• An opinion (MA yes or no) by the CAT has to be agreed by the• Committee for Human Medicines (CHMP; PEI membership).

• MA is granted by the European Commission.

Regulation (EC) No. 1394/2007 (30 Dec. 2008)Regulation (EC) No. 1394/2007 (30 Dec. 2008)A uniform regulatory framework for ATMP in the EUA uniform regulatory framework for ATMP in the EU

Innovation Office

Regulation (EC) No. 1394/2007 Need for central marketing authorisation by end of 2012 at the latest

Article 28 of the Regulation National Regulation „Hospital exemption“

National Implementation in Germany Section 4 b (sub-section 3) of the German Medicinal Products Act

Legal Provision and Starting PointLegal Provision and Starting Point

Innovation Office

• Section 4 b AMG introduced in July 2009 Non-routinely prepared ATMP having characteristics as in Section 4 b (1) require an authorisation by the PEI. Continued use if marketed and used on 23 July 2009:• application to PEI by 01 August 2010 for gene and cell therapy

products• application to PEI by 01 January 2011 for TEP.A decision should be taken within 5 months (clock-stop for answering

questions on possible grounds for non-acceptance).

• Use of ATMP not having a marketing authorisation: Section 40 ff AMG• Clinical trial authorisation by PEI and positive appraisal by ethics

committee.

Regulation on a National LevelRegulation on a National Level

Innovation Office

Decision Tree for Section 4b AMG (German Medicinal Products Act)

http://www.pei.de/cln_170/nn_1743502/SharedDocs/Downloads/pu/innovationsbuero/entscheidungsbaum-_C2_A74b- amg,templateId=raw,property=publicationFile.pdf/entscheidungsbaum-%C2%A74b-amg.pdf

Innovation Office

Co-ordination of national advice,

regulatory and scientificwith focus on ATMP

Preparing and providing (up-dated) information on ATMP

Tasks of the Innovation OfficeTasks of the Innovation Office

Innovation Office

Regulatoryadvice on

technical/strategicquestions

Advice onlicensing

procedures, e.g. national /

european

Advice on specific

regulations, guidelines etc.

Regulatory AdviceRegulatory Advice

Innovation Office

What are the regulatory requirements?What is a possible/best practice procedure – national/European;

„hospital exemption“/clinical trial?When to ask for (national) scientific advice? How to apply for certification/classification with the EMA?

FAQs FAQs -- regulatoryregulatory

Innovation Office

Very early stage of development of themedicinal product Preparing a

clinical trial

Preparing an application of a

clinical trialauthorisation

Preparing an application

for a national authorisation

(Section 4b AMG)

Preparing an application

for a centralisedmarketing

authorisation

Stages for National Scientific AdviceStages for National Scientific Advice

Innovation Office

Provide advice on:

Identification of a risk-based approachDevelopment of medicinal product and proof of

concept/first in manConcept designs of clinical trials /

definition of relevant endpoints

FAQs FAQs -- scientificscientific

Innovation Office

Points of Discussion and RequirementsPoints of Discussion and Requirements

Innovation Office

Haematology / Transfusion Medicine

PEI-ExpertsMedical Biotechnology:

Immunology: Microbial Safety

Clinical Trials

Viral Safety

Advanced Therapy Medicinal Products

Gene Transfer Medicinal Products

Tissue Engineering, Somatic Cell Therapeutics

Therapeutic Vaccines

Biostatistics

Legal Affairs

Pharmakovigilance

EU-Co-operation Biological Medicinal Products

Areas of Advice and ExpertiseAreas of Advice and Expertise

Innovation Office

SummarySummary

1) e.g. genetically modified cells as therapeutic vaccines

2) e.g. autologous haematopoietic bone marrow stem cells for the treatment of myocardial infarction

3) e.g. autologous chondrocyte-transplants for the treatment of cartilage defects

National Advice for ATMP by the Innovation Office of the PEI

First application-oriented development and /

or application

non-clinical studies

clinicaltrials

MAA, European

Supportive process-related advice on the basis of first data throughout the development of the ATMP

------------------------*) Arzneimittelgesetz = German Medicinal Products Act

Co-ordination of scientific advice:

Provision of advice on general regulatory issues

Addressees: Academia (clinical research groups) and companies focused on ATMP

Section 4b AMG*): national

Scientific advice with regardto the requirements of

the quality of ATMP

Scientific advice with regardto preclinical

development of ATMP

Scientific advice with regardto applications

for clinical trials of ATMP

Innovationsbüro

Innovation Office

Workshop for discussion on

common requirements (e.g. advice or product

specific)

Consideration of specific

requirements defined by IQWiG

in PEI- advice procedures

Involvement of IQWiG / G-BA

in commenting minutes of advice

Advice by

G-BA

Consideration of the expertise

of the PEI

Involvement of IQWiG / PEI

in commenting minutes of advice

*Joint Federal Committee ** German Institute for Quality and Efficiency in Healthcare

Innovation Office

ATMPIn accordance with

Regulation (EC) No. 1394/2007,Section 4b AMG,

PEI IQWiG Optional:

involvement byG-BA + PEI

Medicinal productsin accordance with the

AMNOG

G-BA ATMP?

Innovation Office

Demand on advice for scientificrequirements

of clinical trials(PEI)

Demand on advicefor requirements of clincial trials with

regard to (additional) health benefit

(IQWiG)

Advice on the basis of § 35a SGB (social code) V

(G-BA)

Advice on ATMP Basis?

(IQWiG und G-BA + PEI?)

Applicant

Innovation Office

- Central contact point for requests for advice- Co-ordination of advice across various expert areas- Contact point for further information and queries on

planned or completed advice- „All-in-one service“:

bridge to EMA, IQWiG and G-BA and as far as possible to clinical trial centres and non-clinical facilities

Function of the Innovation OfficeFunction of the Innovation Office

Innovation Office

General information on the website of the PEI

Request form for national advice

Booklet on ATMP as guide to applicants

Guides for national applications for ATMP

Register of useful guidelines and links

Selection of FAQs

Service and InformationService and Information

Innovation Office

Some details on advice Some details on advice

Category of the ATMP

Type of Application

of the ATMP

Stage of Development

Innovation Office

100%

22% 19% 25%9%

18%

0%20%40%60%80%

100%201045 =

Main focuses of adviceMain focuses of advice

Innovation Office

Details of advice:Details of advice: QualityQuality

Innovation Office

Details of advice:Details of advice: NonNon--clinicclinic

Innovation Office

Details of advice:Details of advice: ClinicClinic

Innovation Office

Details of advice:Details of advice: Regulatory issuesRegulatory issues

Innovation Office

Advice with regard to product / type of cellsAdvice with regard to product / type of cells

Innovation Office

Advice:Advice: Origin / Type of the cells with regard to indicationOrigin / Type of the cells with regard to indication

45

Innovation Office

Communication with and feedback from stakeholders

Organisation of target group-specific workshops

Organisation of ATMP group-specific workshops

Special workshops on important topics, e.g. special development, bottlenecks

Production and publication of regular printed and/or electronic newsletters, providing overviews of

current scientific issues, developments and trends

Future ProjectsFuture Projects

Innovation Office

Risk-based approach

From VHP to HP

Networks and inclusion of HTAs

Harmonisation of reimbursement

Joint advice

Parallel advice

ChallengesChallenges

Innovation Office

Der Anfang ist die Hälfte des

Ganzen

Aristoteles

Passion is the only orator who can always convince us.

Innovation Office

Dr. Lotte Dahl Tel. 06103/77-2131

Athalia Müller, B.Sc. Tel. 06103/77-1038

Bettina Ziegele, M.A., Head Tel: 06103/77-1012

E-mail: innovation@pei.de

For further information, please click on the following link.:http://www.pei.de/cln_092/nn_154420/DE/infos/pu/innovationsbuero/innovationsbuero-inhalt.html?__nnn=true

ContactContact

>>www.pei.de

Thank you!

Innovation Office

Decision Tree for ATMP

http://www.pei.de/cln_170/nn_1743502/SharedDocs/Downloads/pu/innovationsbuero/entscheidungsbaum- atmp,templateId=raw,property=publicationFile.pdf/entscheidungsbaum-atmp.pdf

Innovation Office

Guide for Application: Module 0

http://www.pei.de/cln_170/nn_1946116/SharedDocs/Downloads/pu/innovationsbuero/modul-0- formulare,templateId=raw,property=publicationFile.pdf/modul-0-formulare.pdf

Innovation Office

Guide for Application: Module 1

http://www.pei.de/cln_170/nn_1946116/SharedDocs/Downloads/pu/innovationsbuero/modul-1- arzneimittelerfassung,templateId=raw,property=publicationFile.pdf/modul-1-arzneimittelerfassung.pdf

Innovation Office

Guide for Application: Modules 3A + 3B

http://www.pei.de/cln_170/nn_1946116/SharedDocs/D ownloads/pu/innovationsbuero/modul-3a- qualitaetsdaten,templateId=raw,property=publicationFi le.pdf/modul-3a-qualitaetsdaten.pdf

http://www.pei.de/cln_170/nn_1946116/SharedDocs/Dow nloads/pu/innovationsbuero/modul-3b- qualitaetsdaten,templateId=raw,property=publicationFile. doc/modul-3b-qualitaetsdaten.doc

Innovation Office

Guide for Application: Module 4

http://www.pei.de/cln_170/nn_1946116/SharedDocs/Downloads/pu/innovationsbuero/modul-4- nichtklinische_20daten,templateId=raw,property=publicationFile.pdf/modul-4-nichtklinische%20daten.pdf

Innovation Office

Guide for Application: Module 5

http://www.pei.de/cln_170/nn_1946116/SharedDocs/Downloads/pu/innovationsbuero/modul-5- klinische_20daten,templateId=raw,property=publicationFile.pdf/modul-5-klinische%20daten.pdf